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CAN-GT rAAV-Olig001-ASPA Gene Therapy in Children With Canavan Disease
2024-519412-13-00Phase 2Authorised
- Sponsor
- Myrtelle
- Start
- Sep 2026
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 2
01
Overview
Phase 2, open-label, single-arm trial of MYR-101 in Canavan disease, sponsored by Myrtelle. Primary endpoint: Change from baseline to Month 6 in CSF NAA concentrations. Planned start Sep 2026; status: authorised.
02
Drugs
MYR-101
03
Conditions
Canavan disease
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